NEW YORK – Durham, North Carolina-based Atsena Therapeutics this week said the US Food and Drug Administration has granted it orphan drug designation for its investigational gene therapy for X-linked retinoschisis, an inherited disease that causes retinal degeneration and vision loss. Atsena is testing the safety and tolerability of the gene therapy, ATSN-201, in male patients at least 6 years of age with the disease caused by mutations in the RS1 gene within the open-label Phase I/II LIGHTHOUSE trial. ATSN-201 previously received rare pediatric disease designation from the FDA.
The US Food and Drug Administration has granted Papillon Therapeutics orphan drug designation for PPL-001, an investigational treatment for Friedreich's ataxia, a rare and inherited neurodegenerative disorder, the San Diego-based company said this week. PPL-001 is a gene-corrected CD34-positive hematopoietic stem and progenitor cell therapy that specifically corrects the GAA repeat expansion in Intron 1 of the FXN gene, a mutation that causes Friedreich's ataxia in many patients with the disease.
City of Hope this week said it received a $150 million gift that it will use to help advance pancreatic cancer research. The donation was given by A. Emmet Stephenson Jr. and his daughter Tessa Stephenson Brand. The funds will be used to start the annual $1 million Stephenson Prize for pancreatic cancer research, establish the Stephenson Fellows Program to provide grants to pancreatic cancer researchers, and provide immediate funding to advance novel immunotherapies and support clinical research in pancreatic cancer. The gift will also be used to create a pancreatic biorepository at City of Hope to support genomics-informed pancreatic cancer patient care such as early detection, research on cancer biology and immune response, and developing novel therapies.
Laboratory Corporation of America said this week that effective Dec. 1, it will be the Blue Cross and Blue Shield of Alabama exclusive national laboratory in the insurer’s Preferred Medical Laboratory Program and the only Preferred Medical Laboratory Plus provider in its outpatient laboratory network. Covered lab services provided by Labcorp and other contracted Preferred Medical Laboratories will be processed at the in-network level of benefits.
The company also announced that it has completed its $237.5 million acquisition of select assets of BioReference Health. Originally announced in March, the acquisition includes BioReference’s laboratory testing businesses focused on clinical diagnostics and reproductive and women's health in the United States outside of New York and New Jersey, including certain patient service centers, customer contracts, and operating assets, which currently generate approximately $100 million in annual revenue.
Quest Diagnostics said this week that it has completed its acquisition of select assets from Allina Health for an undisclosed amount. Under the deal, which was originally announced in June, Quest will offer its laboratory services to Allina Health clinic physicians and outreach providers in Minnesota and western Wisconsin.
The liquid biopsy company Angle said this week that it has inked a deal with Recursion Pharmaceuticals to conduct a pilot study. The firms did not provide further details on the study other than that it will involve using Angle’s Parsortix CTC analysis and that there will be a potential for larger follow-on contracts if the pilot study is successful.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.